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EMBO molecular medicine

Promoterless gene targeting without nucleases rescues lethality of a Crigler-Najjar syndrome mouse model.


PMID 28751579

Abstract

Crigler-Najjar syndrome type I (CNSI) is a rare monogenic disease characterized by severe neonatal unconjugated hyperbilirubinemia with a lifelong risk of neurological damage and death. Liver transplantation is the only curative option, which has several limitations and risks. We applied an